CHMP recommends expanding indication for Hemlibra in haemophilia A.- Roche

Roche announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in adults and children with severe haemophilia A (congenital factor VIII deficiency, FVIII less than 1%) without factor VIII inhibitors. The CHMP has also recommended that Hemlibra can be used at multiple dosing options (once weekly, every two weeks, or every four weeks) for all indicated people with haemophilia A, including those with factor VIII inhibitors.

The positive CHMP opinion is based on results from the pivotal HAVEN 3 and HAVEN 4 studies. In the HAVEN 3 study in people with haemophilia A without factor VIII inhibitors, Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis, and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison. In the HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors, Hemlibra showed a clinically meaningful control of bleeding when dosed every four weeks. Based on this opinion, a final decision regarding the approval of Hemlibra for people without factor VIII inhibitors is expected from the European Commission in the near future.

Comment: Hemlibra was granted Breakthrough Therapy Designation by the FDA for haemophilia A without factor VIII inhibitors. It was also granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease. It was first FDA approved in 2017 and EU approved for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors in February 2018.